Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

• ClinicalTrials.gov Identifier: NCT03500549
• Recruitment Status: Completed
• First Posted: April 18, 2018
• Results First Posted: March 25, 2022
• Last Update Posted: March 25, 2022
• Sponsor: Apellis Pharmaceuticals, Inc.
• Information provided by (Responsible Party): Apellis Pharmaceuticals, Inc.

Study Description

Brief Summary:

Evaluation of the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria

Condition or disease	Intervention/treatment	Phase
Paroxysmal Nocturnal Hemoglobinuria	Drug: Pegcetacoplan; Drug: Soliris	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 80 participants
• Allocation: Randomized
• Intervention Model: Crossover Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Actual Study Start Date: June 14, 2018
• Actual Primary Completion Date: November 14, 2019
• Actual Study Completion Date: August 13, 2020

Arms and Interventions

Arm	Intervention/treatment
Experimental: Pegcetacoplan; 1080 mg pegcetacoplan administered subcutaneously twice-weekly or every three days.	Drug: Pegcetacoplan; Complement (C3) Inhibitor; Other Name: APL-2; Drug: Soliris; Complement (C5) Inhibitor
Active Comparator: Eculizumab; Complement (C5) Inhibitor.	Drug: Pegcetacoplan; Complement (C3) Inhibitor; Other Name: APL-2; Drug: Soliris; Complement (C5) Inhibitor

Outcome Measures

Primary Outcome Measures :

1. Least Squares (LS) Mean Change From Baseline to Week 16 in Hemoglobin (Hb) Level During the RCP [ Time Frame: Baseline and Week 16 ]
   Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions.

Secondary Outcome Measures :

1. Percentage of Subjects Who Did Not Require a Transfusion (Transfusion Avoidance) During the RCP [ Time Frame: Day 1 to Week 16 ]
   Subjects who experienced more than 1 transfusion during the RCP are only counted once. Subjects who did not have a transfusion but withdrew before Week 16 were considered as having a transfusion in the analysis of transfusion avoidance.
2. LS Mean Change From Baseline to Week 16 in Absolute Reticulocyte Count (ARC) During the RCP [ Time Frame: Baseline and Week 16 ]
   Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.
3. LS Mean Change From Baseline to Week 16 in Lactate Dehydrogenase (LDH) Level During the RCP [ Time Frame: Baseline and Week 16 ]
   Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.
4. LS Mean Change From Baseline to Week 16 in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Score During the RCP [ Time Frame: Baseline and Week 16 ]
   The FACIT-fatigue scale version 4 is a 13-item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. A higher score corresponds to a higher quality of life (QoL). Baseline was the last available, nonmissing observation before taking the first dose of pegcetacoplan. Data collected after transfusion is excluded from analysis.
5. Percentage of Subjects Who Achieved a Hb Response in the Absence of Transfusions at Week 16 [ Time Frame: Baseline and Week 16 ]
   Hb response was defined as an increase of at least 1 g/dL in Hb from Baseline at Week 16. Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions.
6. Percentage of Subjects Who Achieved Reticulocyte Normalization in the Absence of Transfusions at Week 16 [ Time Frame: Week 16 ]
   Reticulocyte normalization was defined as the ARC being below the upper limit of the gender-specific normal range at Week 16, censored for transfusions. Subjects who received a transfusion between Day 1 and Week 16 or withdrew without providing efficacy data at Week 16 were classified as nonresponders.
7. Percentage of Subjects Who Achieved Hb Normalization in the Absence of Transfusions at Week 16 [ Time Frame: Week 16 ]
   Hb normalization was defined as the Hb level being above the lower limit of the normal range at Week 16, censored for transfusions. Subjects who received a transfusion between Day 1 and Week 16 or withdrew without providing efficacy data at Week 16 are classified as nonnormalization.
8. LS Mean Change From Baseline to Week 16 in Indirect Bilirubin Level During the RCP [ Time Frame: Baseline and Week 16 ]
   Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.
9. LS Mean Change From Baseline to Week 16 in Haptoglobin Level During the RCP [ Time Frame: Baseline and Week 16 ]
   Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.
10. LS Mean Change From Baseline to Week 16 in Linear Analog Scale Assessment (LASA) Scores During the RCP [ Time Frame: Baseline and Week 16 ]
    The LASA consists of 3 items, where the respondents were asked to rate their perceived level of functioning. Specific domains included activity level, ability to carry out daily activities, and an item for overall QoL. Their level of functioning was reported on a 0 to 100 scale with 0 indicates "As low as could be" and 100 indicates "As high as could be". The combined score ranged from 0 to 300, with higher scores corresponding to a higher QoL.
11. LS Mean Change From Baseline to Week 16 in European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core 30 Scale (QLQ-C30) Scores During the RCP [ Time Frame: Baseline and Week 16 ]
    The EORTC QLQ-C30 questionnaire (version 3.0) consists of 30 questions comprised of both multi-item scales and single-item measures to assess overall QoL in subjects. Questions are designated by functional scales, symptom scales, and global subject QoL/overall perceived health status. For the first 28 questions the 4 possible responses are "Not at all' (1), 'A little' (2), 'Quite a bit' (3) and 'Very much' (4). For the remaining 2 questions the response is requested on a 7-point scale from 1 ('Very poor') to 7 ('Excellent'). The raw scale scores were linear transformed, producing scale scores that ranged from 0% to 100%. A high scale score represents a higher response level. Hence for the functional scales and the global health status a higher score indicates a better QoL, whilst for the symptom scale scores this is implied by a lower score.
12. Total Number of PRBC Units Transfused During the RCP [ Time Frame: Day 1 to Week 16 ]
    Subjects who withdrew during the RCP before Week 16 will have their number of units of PRBC estimated from the duration they were in the study.
13. Mean Change From Baseline to Week 48 in Hb Level During the Treatment Period [ Time Frame: Baseline and Week 48 ]
    Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions.
14. Mean Change From Week 17 to Week 48 in Hb Level During the Open-label Period [ Time Frame: Week 17 and Week 48 ]
    Baseline was the average of measurements recorded before taking the first dose of pegcetacoplan, which included local and central laboratory values during the screening period. Analysis excluded data before the RCP and was censored for transfusions.
15. Mean Change From Baseline to Week 48 in ARC During the Treatment Period [ Time Frame: Baseline and Week 48 ]
    Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.
16. Mean Change From Week 17 to Week 48 in ARC During the Open-label Period [ Time Frame: Week 17 and Week 48 ]
    Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.
17. Mean Change From Baseline to Week 48 in LDH Level During the Treatment Period [ Time Frame: Baseline and Week 48 ]
    Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.
18. Mean Change From Week 17 to Week 48 in LDH Level During the Open-label Period [ Time Frame: Week 17 and Week 48 ]
    Baseline was the average of available measurements recorded from central laboratory before taking the first dose of pegcetacoplan. Analysis excluded data before the RCP and was censored for transfusions.
19. Mean Change From Baseline to Week 48 in FACIT-Fatigue Scale Score During the Treatment Period [ Time Frame: Baseline and Week 48 ]
    The FACIT-fatigue scale version 4 is a 13-item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. A higher score corresponds to a higher QoL. Baseline was the last available, nonmissing observation before taking the first dose of pegcetacoplan. Data collected after transfusion is excluded from analysis.
20. Mean Change From Week 17 to Week 48 in FACIT-Fatigue Scale Score During the Open-label Period [ Time Frame: Week 17 and Week 48 ]
    The FACIT-fatigue scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. Higher score corresponds to a higher QoL.
21. Mean Change From Baseline to Week 48 in LASA Scores During the Treatment Period [ Time Frame: Baseline and Week 48 ]
    The LASA consists of 3 items, where the respondents were asked to rate their perceived level of functioning. Specific domains included activity level, ability to carry out daily activities, and an item for overall QoL. Their level of functioning was reported on a 0 to 100 scale with 0 indicates "As low as could be" and 100 indicates "As high as could be". The combined score ranged from 0 to 300, with higher scores corresponding to a higher QoL.
22. Mean Change From Week 17 to Week 48 in LASA Scores During the Open-label Period [ Time Frame: Week 17 and Week 48 ]
    The FACIT-fatigue scale is a 13 item Likert scaled instrument where the subject was presented with 13 statements and asked to indicate their response as it applied to the past 7 days. The 5 possible responses were 'Not at all' (0), 'A little bit (1), 'Somewhat' (2), 'Quite a bit' (3) and 'Very much' (4). With 13 statements the total score had a range of 0 to 52. Higher score corresponds to a higher QoL.
23. Mean Change From Baseline to Week 48 in QLQ-C30 Scores During the Treatment Period [ Time Frame: Baseline and Week 48 ]
    The EORTC QLQ-C30 questionnaire (version 3.0) consists of 30 questions comprised of both multi-item scales and single-item measures to assess overall QoL in subjects. Questions are designated by functional scales, symptom scales, and global subject QoL/overall perceived health status. For the first 28 questions the 4 possible responses are 'Not at all' (1), 'A little' (2), 'Quite a bit' (3) and 'Very much' (4). For the remaining 2 questions the response is requested on a 7-point scale from 1 ('Very poor') to 7 ('Excellent'). The raw scale scores were linear transformed, producing scale scores that ranged from 0% to 100%. A high scale score represents a higher response level. Hence for the functional scales and the global health status a higher score indicates a better QoL, whilst for the symptom scale scores this is implied by a lower score.
24. Mean Change From Week 17 to Week 48 in QLQ-C30 Scores During the Open-label Period [ Time Frame: Week 17 and Week 48 ]
    The EORTC QLQ-C30 questionnaire (version 3.0) consists of 30 questions comprised of both multi-item scales and single-item measures to assess overall QoL in subjects. Questions are designated by functional scales, symptom scales, and global subject QoL/overall perceived health status. For the first 28 questions the 4 possible responses are 'Not at all' (1), 'A little' (2), 'Quite a bit' (3) and 'Very much' (4). For the remaining 2 questions the response is requested on a 7-point scale from 1 ('Very poor') to 7 ('Excellent'). The raw scale scores were linear transformed, producing scale scores that ranged from 0% to 100%. A high scale score represents a higher response level. Hence for the functional scales and the global health status a higher score indicates a better QoL, whilst for the symptom scale scores this is implied by a lower score.
25. Total Number of PRBC Units Transfused During the Open-Label Period [ Time Frame: Week 17 to Week 48 ]
    Number of units of PRBC transfused to subjects in the open-label period are reported.

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• At least 18 years of age
• Primary diagnosis of PNH confirmed by high-sensitivity flow cytometry
• On treatment with eculizumab. Dose of eculizumab must have been stable for at least 3 months prior to the Screening Visit
• Hb <10.5 g/dL at the Screening Visit
• Absolute reticulocyte count > 1.0x ULN at the Screening Visit
• Platelet count of >50,000/mm3 at the Screening Visit
• Absolute neutrophil count >500/mm3 at the Screening Visit
• Vaccination against Neisseria meningitides types A, C, W, Y and B, Streptococcus pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day 1 dosing, or within 14 days after starting treatment with APL-2. Unless documented evidence exists that subjects are non-responders to vaccination as evidenced by titers or display titer levels within acceptable local limits
• Women of child-bearing potential (WOCBP) must have a negative pregnancy test at the Screening and Day -28 Visit (Run-in Period) and must agree to use protocol defined methods of contraception for the duration of the study and 90 days after their last dose of study drug
• Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study and 90 days after their last dose of study drug
• Willing and able to give informed consent
• Willing and able to self-administer APL-2 (administration by caregiver will be allowed)
• Have a body mass index (BMI) ≤35.0 kg/m2

Exclusion Criteria:

• Active bacterial infection that has not resolved within 14 week of Day -28 (first dose of APL-2)
• Receiving iron, folic acid, vitamin B12 and EPO, unless the dose is stable, in the 4 weeks prior to Screening
• Hereditary complement deficiency
• History of bone marrow transplantation
• History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration
• Participation in any other investigational drug trial or exposure to other investigational agent within 30 days or 5 half-lives (whichever is longer)
• Currently breast-feeding women
• Inability to cooperate or any condition that, in the opinion of the investigator, could increase the subject's risk of participating in the study or confound the outcome of the study

This study includes cardiac safety evaluations. The following cardiac eligibility criteria are necessary to avoid confounding the cardiac safety outcomes:

• History or family history of Long QT Syndrome or torsade de pointes, unexplained syncope, syncope from an uncorrected cardiac etiology, or family history of sudden death
• Myocardial infarction, CABG, coronary or cerebral artery stenting and /or angioplasty, stroke, cardiac surgery, or hospitalization for congestive heart failure within 3 months or greater than Class 2 Angina Pectoris or NYHA Heart Failure Class >2
• QTcF > 470 ms, PR > 280 ms
• Mobitz II 2nd degree AV Block, 2:1 AV Block, High Grade AV Block, or Complete Heart Block unless the patient has an implanted pacemaker or implantable cardiac defibrillator (ICD) with backup pacing capabilities
• Receiving Class 1 or Class 3 antiarrhythmic agents, or arsenic, methadone, ondansetron or pentamidine at screening
• Receiving any other QTc-prolonging drugs (see Appendix 4 in Section 19.4), at a stable dose for less than 3 weeks prior to dosing
• Receiving prophylactic ciprofloxacin, erythromycin or azithromycin for less than one week prior to the first dose of study medication (must have a repeat screening ECG after one week of prophylactic antibiotics with QTcF < 470 ms)

Contacts and Locations

Locations

United States, California

University of Southern California

Los Angeles, California, United States, 90033

Sarcoma Oncology Research Center

Santa Monica, California, United States, 90403

United States, Colorado

Denver Health

Denver, Colorado, United States, 80204

United States, District of Columbia

Georgetown University Hospital

Washington, District of Columbia, United States, 20057

United States, Florida

Cancer Specialists of North Florida

Jacksonville, Florida, United States, 32256

Lakes Research

Miami Lakes, Florida, United States, 33014

Mid Florida Hematology and Oncology

Orange City, Florida, United States, 32763

United States, Georgia

Winship Cancer Institute of Emory University

Atlanta, Georgia, United States, 30322

United States, Illinois

Northwestern University

Chicago, Illinois, United States, 60611

United States, Indiana

Investigative Clinical Research of Indiana

Indianapolis, Indiana, United States, 46260

United States, Michigan

Cancer & Hematology Centers of Western Michigan

Grand Rapids, Michigan, United States, 49503

United States, New York

New York Cancer & Blood Specialists

Bronx, New York, United States, 10469

Roswell Park Cancer Institute

Buffalo, New York, United States, 14263

New York Cancer & Blood Specialists

East Setauket, New York, United States, 11733

United States, North Carolina

Duke University Medical Center

Durham, North Carolina, United States, 27710

United States, Oregon

Good Samaritan Hospital

Corvallis, Oregon, United States, 97330

United States, Tennessee

University of Tennessee Medical Center

Knoxville, Tennessee, United States, 37920

Baptist Cancer Center

Memphis, Tennessee, United States, 38120

United States, Texas

HOPE Cancer Center of East Texas

Tyler, Texas, United States, 75701

Australia, Victoria

Royal Melbourne Hospital

Melbourne, Victoria, Australia, 3000

Belgium

Cliniques Universitaires Saint-Luc

Woluwe-Saint-Lambert, Vlaams-Brabant, Belgium, 1200

AZ Delta Campus Wilgenstraat

Roeselare, West-Vlaanderen, Belgium, 8800

Canada, Alberta

University of Alberta

Edmonton, Alberta, Canada, T6G 2G3

Canada, Ontario

Toronto General Hospital

Toronto, Ontario, Canada, M9A1G2

France

Centre Hospitalier Annecy Genevois

Annecy, France, 74374

Centre Hospitalier William Morey

Chalon-sur-Saône, France, 71100

Centre Hospitalier Universitaire de Lille

Lille, France, 59037

Institut Paoli-Calmettes Marseille

Marseille, France, 13009

Hôpital Saint-Louis

Paris, France, 75010

Centre Hospitalier Lyon Sud

Pierre-Bénite, France, 69495

Centre Hospitalier de Saint-Quentin

Saint-Quentin, France, 02321

Institut Universitaire du Cancer Toulouse - Oncopole

Toulouse, France, 31059

Germany

Universitätsklinikum Ulm

Ulm, Baden-Württemberg, Germany, 89081

Uniklinik RWTH Aachen

Aachen, North Rhine-Westphalia, Germany, 52074

Universitätsklinikum Essen

Essen, North Rhine-Westphalia, Germany, 45147

Universitätsklinikum Hamburg Eppendorf

Hamburg, Germany, 20246

Japan

Japanese Red Cross Nagoya Daiichi Hospital

Nagoya, Aichi, Japan, 453-8511

Japanese Red Cross Nagoya Daini Hospital

Showa-ku, Aichi, Japan, 453-8650

University of Tsukuba Hospital

Tsukuba, Ibaraki, Japan, 305-8576

Shinshu University Hospital

Matsumoto, Nagano, Japan, 390-8621

Okayama University Hospital

Okayama-shi, Okayama, Japan, 700-8558

Juntendo University Hospital

Bunkyo-ku, Tokyo, Japan, 113-8421

NTT Medical Center Tokyo

Shinagawa-ku, Tokyo, Japan, 141-8625

Kinan Hospital

Tanabe, Wakayama, Japan, 646-8588

Korea, Republic of

Soonchunhyang University Bucheon Hospital

Bucheon, Korea, Republic of, 14584

Chungnam National University Hospital

Daejeon, Korea, Republic of, 35015

Samsung Medical Center

Seoul, Korea, Republic of, 06351

Russian Federation

Pavlov First Saint Petersburg State Medical University of Russian Ministry of Health

Saint Petersburg, Russian Federation, 197022

Pavlov First Saint Petersburg State Medical University

Saint Petersburg, Russian Federation, 197022

Institution of Health Care of Tyumen Region

Tyumen, Russian Federation, 625023

Spain

Hospital Universitario de Gran Canaria Dr. Negrín

Las Palmas De Gran Canaria, Spain, 35010

Hospital Universitario Politécnico La Fe

Valencia, Spain, 46026

United Kingdom

St. James' Institute of Oncology, Leeds Teaching Hospitals

Leeds, United Kingdom, LS9 7TF

Sponsors and Collaborators

Apellis Pharmaceuticals, Inc.

  Study Documents (Full-Text)

Documents provided by Apellis Pharmaceuticals, Inc.:

Study Protocol  [PDF] August 16, 2019

Statistical Analysis Plan  [PDF] December 5, 2019

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

de Latour RP, Szer J, Weitz IC, Roth A, Hochsmann B, Panse J, Usuki K, Griffin M, Kiladjian JJ, de Castro CM, Nishimori H, Ajayi T, Al-Adhami M, Deschatelets P, Francois C, Grossi F, Risitano AM, Hillmen P. Pegcetacoplan versus eculizumab in patients with paroxysmal nocturnal haemoglobinuria (PEGASUS): 48-week follow-up of a randomised, open-label, phase 3, active-comparator, controlled trial. Lancet Haematol. 2022 Sep;9(9):e648-e659. doi: 10.1016/S2352-3026(22)00210-1.

Cella D, Sarda SP, Hsieh R, Fishman J, Hakimi Z, Hoffman K, Al-Adhami M, Nazir J, Cutts K, Lenderking WR. Changes in hemoglobin and clinical outcomes drive improvements in fatigue, quality of life, and physical function in patients with paroxysmal nocturnal hemoglobinuria: post hoc analyses from the phase III PEGASUS study. Ann Hematol. 2022 Sep;101(9):1905-1914. doi: 10.1007/s00277-022-04887-8. Epub 2022 Jul 23.

Hakimi Z, Wilson K, McAughey E, Pochopien M, Wojciechowski P, Toumi M, Knight C, Sarda SP, Patel N, Wiseman C, de Castro NP, Nazir J, Kelly RJ. The cost-effectiveness, of pegcetacoplan compared with ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria, in a UK setting. J Comp Eff Res. 2022 Sep;11(13):969-985. doi: 10.2217/cer-2022-0076. Epub 2022 Jul 7.

Bhak RH, Mody-Patel N, Baver SB, Kunzweiler C, Yee CW, Sundaresan S, Swartz N, Duh MS, Krishnan S, Sarda SP. Comparative effectiveness of pegcetacoplan versus ravulizumab in patients with paroxysmal nocturnal hemoglobinuria previously treated with eculizumab: a matching-adjusted indirect comparison. Curr Med Res Opin. 2021 Nov;37(11):1913-1923. doi: 10.1080/03007995.2021.1971182. Epub 2021 Sep 3.

Hillmen P, Szer J, Weitz I, Roth A, Hochsmann B, Panse J, Usuki K, Griffin M, Kiladjian JJ, de Castro C, Nishimori H, Tan L, Hamdani M, Deschatelets P, Francois C, Grossi F, Ajayi T, Risitano A, Peffault de Latour R. Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 2021 Mar 18;384(11):1028-1037. doi: 10.1056/NEJMoa2029073. Erratum In: N Engl J Med. 2024 Mar 14;390(11):1060.

• Responsible Party: Apellis Pharmaceuticals, Inc.
• ClinicalTrials.gov Identifier: NCT03500549
• Other Study ID Numbers: APL2-302
• First Posted: April 18, 2018
• Results First Posted: March 25, 2022
• Last Update Posted: March 25, 2022
• Last Verified: March 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Hemoglobinuria; Hemoglobinuria, Paroxysmal; Proteinuria; Urination Disorders; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Urological Manifestations; Anemia, Hemolytic; Anemia; Hematologic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases; Eculizumab; Complement Inactivating Agents; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs